A prospective natural history study of type 1 spinal muscular atrophy

Enrico Bertini; Eugenio Maria Mercuri

doi:10.1038/nrneurol.2017.189

A prospective natural history study of type 1 spinal muscular atrophy

Enrico Bertini^*
, Eugenio Maria Mercuri

^*Autore corrispondente per questo lavoro

IRCCS Ospedale pediatrico Bambino Gesù - Roma

Risultato della ricerca: Contributo in rivista › Articolo

4 Citazioni (Scopus)

Abstract

Effective drug development for infantile-onset spinal muscular atrophy (SMA) requires a meaningful understanding of disease progression and reliable biomarkers. A new report presents the results of a longitudinal, multicentre, prospective natural history study of SMA, which are critical for the research of future therapies.

Lingua originale	Inglese
pagine (da-a)	197-198
Numero di pagine	2
Rivista	Nature Reviews Neurology
Volume	14
Numero di pubblicazione	4
DOI	https://doi.org/10.1038/nrneurol.2017.189
Stato di pubblicazione	Pubblicato - 2018

All Science Journal Classification (ASJC) codes

Neurologia (clinica)
Neuroscienze Cellulari e Molecolari

Keywords

Cellular and Molecular Neuroscience
Neurology (clinical)

Accesso al documento

10.1038/nrneurol.2017.189

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title = "A prospective natural history study of type 1 spinal muscular atrophy",

abstract = "Effective drug development for infantile-onset spinal muscular atrophy (SMA) requires a meaningful understanding of disease progression and reliable biomarkers. A new report presents the results of a longitudinal, multicentre, prospective natural history study of SMA, which are critical for the research of future therapies.",

keywords = "Cellular and Molecular Neuroscience, Neurology (clinical), Cellular and Molecular Neuroscience, Neurology (clinical)",

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AB - Effective drug development for infantile-onset spinal muscular atrophy (SMA) requires a meaningful understanding of disease progression and reliable biomarkers. A new report presents the results of a longitudinal, multicentre, prospective natural history study of SMA, which are critical for the research of future therapies.

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KW - Neurology (clinical)

KW - Cellular and Molecular Neuroscience

KW - Neurology (clinical)

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A prospective natural history study of type 1 spinal muscular atrophy

Abstract

All Science Journal Classification (ASJC) codes

Keywords

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